Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature
Aleena Khan, Laura E. Case, Mrudu Herbert, Stephanie DeArmey, Harrison N. Jones, Kelly D. Crisp, Kanecia O. Zimmerman, Mai K. ElMallah, Sarah P. Young, Priya S. Kishnani
Topics & Concepts
Enzyme replacement therapyMedicineDosingInternal medicineCreatine kinaseDiseaseCreatinePulmonary function testingPlaceboGlycogen storage disease type IIGastroenterologyPediatricsPathologyAlternative medicineLysosomal Storage Disorders ResearchGlycogen Storage Diseases and MyoclonusWhipple's Disease and Interleukins